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Name:
UMIN ID:

Recruitment status No longer recruiting
Unique ID issued by UMIN UMIN000028429
Receipt No. R000032495
Scientific Title Multicenter randomized double-blind comparison test followed by open-label continuous administration test of NPC-12T for Fibrodysplasia Ossificans Progressiva
Date of disclosure of the study information 2017/08/01
Last modified on 2018/05/29

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Basic information
Public title Multicenter randomized double-blind comparison test followed by open-label continuous administration test of NPC-12T for Fibrodysplasia Ossificans Progressiva
Acronym Investigator-initiated clinical trial of NPC-12T for Fibrodysplasia Ossificans Progressiva
Scientific Title Multicenter randomized double-blind comparison test followed by open-label continuous administration test of NPC-12T for Fibrodysplasia Ossificans Progressiva
Scientific Title:Acronym Investigator-initiated clinical trial of NPC-12T for Fibrodysplasia Ossificans Progressiva
Region
Japan

Condition
Condition Fibrodysplasia Ossificans Progressiva
Classification by specialty
Pediatrics Orthopedics Rehabilitation medicine
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 To investigate the clinical efficacy and safety of NPC-12T for Fibrodysplasia Ossificans Progressiva by multicenter randomized double-blind placebo-controlled comparison test followed by open-label continuous administration test
Basic objectives2 Safety,Efficacy
Basic objectives -Others
Trial characteristics_1 Exploratory
Trial characteristics_2 Others
Developmental phase Phase II,III

Assessment
Primary outcomes Objective evaluation of physical function using J-HAQ (or J-CHAQ) at the end of double-blind stage
Key secondary outcomes Frequency and grade of adverse events and side effects
Quantitative assessment of heterotopic bone by whole-body CT
Number of newly formed heterotopic bone
Number of flare-up episode
Assessment by CAJIS

Base
Study type Interventional

Study design
Basic design Parallel
Randomization Randomized
Randomization unit Individual
Blinding Double blind -all involved are blinded
Control Placebo
Stratification YES
Dynamic allocation YES
Institution consideration Institution is not considered as adjustment factor.
Blocking YES
Concealment Central registration

Intervention
No. of arms 2
Purpose of intervention Treatment
Type of intervention
Medicine
Interventions/Control_1 Initial dose is 1 mg per day for patients with body surface area less than 1.5 m2, or 2 mg per day for patients with that not less than 1.5 m2. Oral intake will be adjusted targeting trough value between 5 and 15 ng/ml. Maximum dose is 4 mg per day.
Interventions/Control_2 Placebo-control. For placebo group, oral intake will be adjusted randomly.
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit
6 years-old <=
Age-upper limit
59 years-old >=
Gender Male and Female
Key inclusion criteria Patients diagnosed as "Definitive" or "Probable" according to the diagnostic criteria proposed by the research group for intractalble diseases organized by MHLW.
Key exclusion criteria 1. Patients with acute or chronic infection
2. Patients with skin sore reaching dermis
3. Patients experiencing flare-up during last 90 days
4. Past usage of mTOR inhibitors or other molecular target drugs relating mTOR pathway
5. Usage of medicine or foods which are prohibited for concurrent use of Sirolimus
6. History of allergy to Sirolimus, Sirolimus derivatives or additive substance
7. Patients with HBV, with the history of HBV or with active type-C hepatitis.
8. Severe abnormality in blood or liver function
9. Uncontrollable abnormal lipid metabolism
10. Renal failure
11. Immunodeficiency including HIV or primary immunodeficiency
12. Patients who are not able to take tablet, or who have gastrointestinal disorders possibly leading to insufficient absorption of Sirolimus
13. Patients who had surgery during 8 weeks before the registry
14. Pregnant, probably pregnant, or breast-feeding women. Patients who do not agree birth control during clinical trial.
Target sample size 20

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Junya Toguchida
Organization Institute for Frontier Life and Medical Sciences, Kyoto University
Division name Department of Regeneration Science and Engineering, Laboratory of Tissue Regeneration
Zip code
Address 53, Shogoin-Kawahara-cho, Sakyo-ku, Kyoto
TEL 075-751-4134
Email togjun@infront.kyoto-u.ac.jp

Public contact
Name of contact person
1st name
Middle name
Last name Junya Toguchida
Organization Institute for Frontier Life and Medical Sciences, Kyoto University
Division name Department of Regeneration Science and Engineering, Laboratory of Tissue Regeneration
Zip code
Address 53, Shogoin-Kawahara-cho, Sakyo-ku, Kyoto
TEL 075-751-4134
Homepage URL
Email togjun@infront.kyoto-u.ac.jp

Sponsor
Institute Kyoto University
Institute
Department

Funding Source
Organization Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization Japanese Governmental office
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization
Address
Tel
Email

Secondary IDs
Secondary IDs NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions 京都大学医学部附属病院(京都府)
東京大学医学部附属病院(東京都)
名古屋大学医学部附属病院(愛知県)
九州大学病院(福岡県)

Other administrative information
Date of disclosure of the study information
2017 Year 08 Month 01 Day

Related information
URL releasing protocol
Publication of results Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status No longer recruiting
Date of protocol fixation
2017 Year 06 Month 23 Day
Date of IRB
Anticipated trial start date
2017 Year 09 Month 07 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2017 Year 07 Month 28 Day
Last modified on
2018 Year 05 Month 29 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000032495

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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